哮喘联盟

   摘要
   引言：对哮喘症状的评估以及急救药物的使用情况是临床试验中评价哮喘治疗效果的良好研究终点。
   目的：评估哮喘症状量化指标的以及急救药物使用（ASRMU）日志在临床试验中对12岁及以上哮喘患者的的应用。
   方法：与35名患者进行面谈，并告知其ASRMU日志中基本概念的重要性。临床症状的评分以及急救药物的使用用无症状天数（SFD）以及不使用急救药物的天数（RFD）表示。纸笔记录和用电子日记记录两组间的再调查信度以及等效值（基于组内相关系数ICC）用47例患者的前瞻性研究来评估。ASRMU日志的效应通过在八项评估哮喘患者单独或联合使用吸入性皮质类固醇激素（ICS）以及长效β2受体激动剂（LABA）治疗效果的临床试验中治疗组无症状天数及不使用急救药物天数比例的不同来评估。用第9个安慰剂对照研究计算效应值。最小重要差异（MID）通过来自2项临床试验的效标以及与11名患者面谈而确定。
   结果：与患者的面谈支持了ASEMU日志内容的有效性。对于无症状天数的再调查信度是可接受的（ICC：0.70-0.75），但是对于不使用急救药物的天数，其变异较大（ICC：0.58－0.78）。纸笔记录以及用电子日志记录两种管理模式是等效的（SFD，ICC＝ 0.84； RFD， ICC＝ 0.70）。同时使用吸入性皮质类固醇激素以及长效β2受体激动剂组的无症状天数以及不使用急救药物的天数是最多的，其次是单独使用一种治疗药物组，再次是安慰剂组。无症状天数的MID为7.7-14.7%，不使用急救药物天数的MID为8.4-15.6%。
   结论：ASRMU日志可记录对哮喘患者非常重要的疾病特异的相关概念，并为哮喘的诊断以及治疗效果的评估提供重要的信息。
 

（杨冬 审校）
J Asthma. 2014 Sep 10:1-10. [Epub ahead of print]

 

Measurement properties of an asthma symptom and rescue medication use diary.
 

Clark M1, Martin S, Svedsater H, Dale P, Jacques L.
 

ABSTRACT
INTRODUCTION: Assessment of symptoms and rescue medication use are well-established endpoints for clinical trials evaluating asthma treatment.
OBJECTIVE: To evaluate the measurement properties of an asthma symptom and rescue medication use (ASRMU) diary for clinical trials involving asthma patients aged ≥12 years.
METHODS: Interviews with 35 patients were conducted to confirm the importance of key concepts in the ASRMU diary. Scores of symptom and rescue medication use were converted to symptom-free days (SFD) and rescue-free days (RFD). Test-retest reliability and equivalence (based on intra-class correlation coefficients [ICCs]) between paper-and-pencil and electronic (eDiary) versions were evaluated in a prospective study in 47 patients. Responsiveness of the ASRMU diary was evaluated through differences in percentage of SFD and of RFD by treatment group in eight asthma clinical trials that assessed inhaled corticosteroids (ICS) and long-acting β2-agonists (LABA), alone or in combination. A ninth placebo-controlled study calculated effect sizes. Minimal important differences (MID) were determined using anchor-based methods from two trials and by interviewing 11 patients.
RESULTS: Patient interviews supported content validity for the ASRMU diary. Test-retest reliability was acceptable for SFD (ICC:0.70-0.75), but varied for RFD (ICC:0.58-0.78). Paper-and-pencil and eDiary modes of administration were equivalent (SFD, ICC = 0.84; RFD, ICC = 0.70). ICS/LABA had the largest percentage of SFD and RFD, followed by monotherapy and then placebo. MIDs were 7.7-14.7% for SFD and 8.4-15.6% for RFD.
CONCLUSIONS: The ASRMU diary captures the disease-specific concepts of greatest importance to asthma patients and provides important information for asthma diagnosis and treatment evaluation.
 

J Asthma. 2014 Sep 10:1-10. [Epub ahead of print]